Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting

Opus Genetics (Nasdaq: IRD) reported 3-month sentinel participant data for its Phase 1/2 OPGx-BEST1 gene therapy for BEST1-related retinal disease.

Rhea-AI Summary

Opus Genetics (Nasdaq: IRD) reported 3-month sentinel participant data for its Phase 1/2 OPGx-BEST1 gene therapy for BEST1-related retinal disease.

The treated 63-year-old participant showed no ocular inflammation, no treatment-related adverse events, and no dose-limiting toxicities. Efficacy signals included a 12-letter BCVA gain and 23% central subfield thickness (CST) reduction at three months. Full Cohort 1 data expected mid-year 2026.

Positive

• 12-letter BCVA gain in treated eye at three months
• 23% CST reduction in treated eye at three months
• No ocular inflammation or treatment-related adverse events observed
• Resolution of intraretinal fluid seen as early as one month

Negative

• Only the sentinel participant reported; n=1 for presented data
• Full Cohort 1 results not available until mid-year 2026

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Details

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On Feb 27, the day this news came out, IRD closed 15.88% above the previous close.

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Key Figures

BCVA improvement: 12-letter gain CST reduction: 23% decrease Follow-up duration: 3 months +4 more

BCVA improvement

12-letter gain

Best Corrected Visual Acuity in treated eye at three months

CST reduction

23% decrease

Central subfield thickness in treated eye at three months

Follow-up duration

3 months

Sentinel participant OPGx-BEST1 safety and activity readout

Early signal timing

1 month

Earliest observed structural and functional improvements

Participant age

63-year-old

Sentinel Autosomal-Recessive Bestrophinopathy patient

Participants enrolled

2 participants

Phase 1/2 OPGx-BEST1 study enrollment to date

Cohort 1 data timing

3-month results mid-year 2026

Full Cohort 1 OPGx-BEST1 readout guidance

Previous Clinical trial Reports

5 past events · Latest: Feb 25

Same Type 5 events

1. Feb 25

   sNDA acceptance

   24h Move

   -3.9%

   FDA accepted supplemental NDA for presbyopia therapy after successful Phase 3 data.

2. Jan 27

   Trial initiation

   24h Move

   +0.7%

   Launch of OPGx-MERTK gene therapy trial for retinitis pigmentosa with external funding.

3. Dec 09

   DMC recommendation

   24h Move

   +4.4%

   Independent Data Monitoring Committee backed continuation of Phase 1/2 BEST1 trial.

4. Nov 13

   First patient dosed

   24h Move

   -3.6%

   First participant dosed in OPGx-BEST1 Phase 1/2 gene therapy study for Best disease.

5. Sep 30

   Pediatric data

   24h Move

   -7.8%

   Positive three‑month pediatric data from OPGx-LCA5 Phase 1/2 trial in LCA5.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

phase 1/2, gene therapy, subretinal administration, autosomal-recessive bestrophinopathy, +3 more

7 terms

phase 1/2 medical

"ongoing Phase 1/2 study of OPGx-BEST1 gene therapy"

Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.

gene therapy medical

"a clinical-stage biopharmaceutical company developing gene therapies to restore vision"

Gene therapy is a medical technique that involves altering or replacing faulty genes in a person's cells to treat or prevent disease. It is considered a promising area of innovation because it has the potential to provide long-term or even permanent solutions to genetic conditions. For investors, advancements in gene therapy can signal opportunities in biotech companies and emerging treatments with significant growth potential.

subretinal administration medical

"following subretinal administration of OPGx-BEST1"

Subretinal administration is a surgical method that delivers a drug, gene therapy, or cells directly into the space beneath the retina at the back of the eye, placing treatment where the cells that support vision live. It matters to investors because this highly targeted approach can improve effectiveness and limit side effects compared with less direct dosing, but it also increases development complexity, requires specialist surgery and training, and can raise regulatory and commercial risk profiles.

autosomal-recessive bestrophinopathy medical

"a 63-year-old female with Autosomal-Recessive Bestrophinopathy (ARB) disease"

A rare inherited eye disorder caused when a person inherits two faulty copies of the bestrophin gene, leading to abnormal fluid and function in the light-sensing layer at the back of the eye and progressive central vision loss. Investors should care because it creates a defined patient group and unmet medical need that can drive demand for diagnostic tests, treatments or gene therapies; think of it as a small, specific market niche with high clinical and commercial focus.

best corrected visual acuity medical

"an equivalent 12-letter gain in Best Corrected Visual Acuity (BCVA)"

Best corrected visual acuity (BCVA) is the sharpest level of sight a person can reach when using the optimal prescription lenses or other standard corrections, typically measured with an eye chart. For investors, BCVA is a common, standardized outcome in trials and product tests—like checking a camera’s clarity after fine-tuning—so changes in BCVA signal whether an eye treatment or device is delivering real, measurable benefit.

central subfield thickness medical

"structural improvement in central subfield thickness (CST) was observed"

Central subfield thickness is a measurement of the retina’s thickness at the very center of the macula, typically taken by an imaging scan of the eye; think of it like measuring the height of the ground at the center of a small target. It matters to investors because changes in this number are a common, objective medical endpoint used to judge whether eye drugs or devices relieve swelling and improve vision, which influences regulatory approval, market potential, and sales forecasts.

intraretinal fluid medical

"Resolution of intraretinal fluid was also seen as early as 1-month"

Intraretinal fluid is fluid that collects inside the layers of the retina, the light‑sensitive tissue at the back of the eye, caused by leaking blood vessels or inflammation. It is a direct sign of worsening vision conditions and is routinely measured in clinical exams and trials; like water trapped between wallpaper layers, its presence and change over time can determine whether a treatment is seen as effective and therefore drive regulatory decisions, adoption and commercial value for therapies and devices.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting toxicities at three months 
  
  
• Early signals of functional and structural improvement observed at one month and three months
  
  
• 12-letter BCVA gain and 23% CST reduction observed in the treated eye at three months
  
  
• Full cohort data expected in mid-year 2026
  
  

RESEARCH TRIANGLE PARK, N.C., Feb. 27, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), announced today new clinical data from its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy, presented at the 49th Annual Meeting of the Macula Society, in San Diego, California.

The presentation, titled “Preliminary Results from an Adult Participant in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1 Mutations,” reported 3-month results from the first (sentinel) adult participant treated in the study, highlighting positive safety, tolerability, and biological activity following subretinal administration of OPGx-BEST1.

The sentinel participant is a 63-year-old female with Autosomal-Recessive Bestrophinopathy (ARB) disease with severe functional impairment. The data demonstrated that OPGx-BEST1 was well tolerated with no ocular inflammation, no ocular or treatment-related adverse events, and no dose limiting toxicities. Early signals of functional vision improvement were observed, including an equivalent 12-letter gain in Best Corrected Visual Acuity (BCVA) in the treated study eye. In addition, structural improvement in central subfield thickness (CST) was observed with a 23% decrease in the study eye. Resolution of intraretinal fluid was also seen as early as 1-month in areas with less atrophy.

“We are encouraged by these results from our sentinel participant, showing OPGx-BEST1 was well-tolerated and demonstrated promising initial efficacy at three months,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. Although early, this data represents an important milestone for our OPGx-BEST1 program and for patients with BEST1-related retinal diseases.”

“BEST1-related retinal diseases represent a significant unmet medical need, with no approved treatments currently available,” said Mark Pennesi, M.D., Ph.D., study investigator at the Retina Foundation of the Southwest in Dallas, Texas. “The preliminary results from this study, including the early favorable safety profile and initial signals of functional and structural improvement, are encouraging and support continued evaluation of OPGx-BEST1 as a gene augmentation approach for patients with BEST1-associated disease.”

Recruitment in the Phase 1/2 study is ongoing at two clinical sites in the U.S., with additional sites expected to open in Florida, Cincinnati and New York. Two participants have been enrolled to date, with 3-month results from the full Cohort 1 expected in mid-year 2026.

The full presentation and video recording will be available on the Opus Genetics website in the Events section.

About OPGx-BEST1 and the Phase 1/2 Trial

OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models.

By restoring BEST1 function, the therapy aims to address the underlying genetic cause of retinal degeneration and support preservation of photoreceptor health and visual function. BEST1-associated IRDs affect an estimated 22,000 patients worldwide and currently have no approved treatments.

The ongoing adaptive, open-label Phase 1/2 study is evaluating single-eye subretinal administration of OPGx-BEST1 up to two dose levels in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Treatment will be administered via a single subretinal injection in one eye of each participant with two dosing cohorts.

The primary objective is to assess safety and tolerability and identify the most appropriate dose for further clinical development, with participants followed longitudinally for long-term outcomes. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.

About Opus Genetics

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RHO, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional potential indications in presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.   

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements related to the clinical development, clinical results, preclinical data, and future plans for Phentolamine Ophthalmic Solution 0.75%, OPGx-LCA5, OPGx-BEST1, RDH12, and earlier stage programs, and expectations regarding us, our business prospects, and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, our subsequent Quarterly Reports on Form 10-Q, and in our other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “strive,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.

Contacts:

Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com

Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com

Source: Opus Genetics, Inc.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did Opus Genetics (IRD) report for the 3-month OPGx-BEST1 sentinel participant on Feb 27, 2026?

The sentinel participant showed no ocular inflammation, no treatment-related adverse events, and no dose-limiting toxicities. According to Opus Genetics, early efficacy signals included a 12-letter BCVA gain and 23% CST reduction in the treated eye at three months.

How meaningful is a 12-letter BCVA gain reported by Opus Genetics (IRD) at three months?

A 12-letter BCVA gain indicates a notable functional vision improvement in the treated eye within three months. According to Opus Genetics, this was observed in the sentinel adult participant and represents an early efficacy signal requiring confirmation in the full cohort.

When will Opus Genetics (IRD) release full Cohort 1 results for OPGx-BEST1?

Full Cohort 1 results are expected in mid-year 2026, per the company announcement. According to Opus Genetics, recruitment is ongoing and additional U.S. sites are planned to open before the full cohort readout.

What safety findings did Opus Genetics (IRD) present for OPGx-BEST1 at three months?

The company reported no ocular inflammation, no ocular or treatment-related adverse events, and no dose-limiting toxicities. According to Opus Genetics, these safety signals come from the sentinel participant at the three-month assessment.

How did OPGx-BEST1 affect retinal structure in the reported patient at three months?

Structural improvement included a 23% reduction in central subfield thickness and resolution of intraretinal fluid in some areas. According to Opus Genetics, these changes were seen in the treated eye at one and three months, indicating early anatomical response.